Compassionate Drug Use

What is compassionate drug use?

Compassionate drug use (or sometimes just compassionate use) is the use of a new, unapproved drug to treat a seriously ill patient when no other treatments are available.

Drugs that are being tested but have not yet been approved by the US Food and Drug Administration (FDA) are called investigationaldrugs. These drugs are normally available only to people who are taking part in a clinical trial (a research study that is testing the drug).

Being able to use one of these drugs when you are not in a clinical trial is most commonly referred to as compassionate drug use.

Is compassionate drug use legal?

Compassionate drug use is legal, but it’s tightly restricted to people who meet certain conditions.

Who might benefit from compassionate drug use?

Patients with serious or life-threatening conditions who aren't able to get access to an unapproved drug through a clinical trial might benefit from compassionate use, if it's available.

How are unapproved drugs accessed through compassionate drug use?

The simplest way to get an unapproved drug is through a clinical trial. But many people with life-threatening diseases can't find suitable clinical trials. This might be because they live too far from cancer research centers where the studies are being done, or because they're not eligible for some reason.

People who aren’t in clinical trials might be able to get access to an unapproved drug from the company that makes it in 2 ways:

Through expanded access programs (EAPs)

Through "Right to Try"

Here are some important things to know about drugs that may be available through compassionate use:

Not all drugs are available for compassionate use, and drug companies aren't required to supply their drugs through compassionate use. Drug companies have different policies and processes, but laws require all drug developers to publicly post contact information for compassionate use requests.

There may be very limited amounts of a drug available for compassionate use, or drug companies might only have enough drug for use in clinical trials. (Producing extra medicine for people who are not in clinical trials can be costly for the drug company, especially when there's a change the drug might never be approved.)

Some drug companies will supply a drug through compassionate use for free, but others might charge patients. Most insurance companies will not pay for the costs of the investigational drugs themselves, even with compassionate use. There might also be other costs, such as the clinic's charges for giving the drug and monitoring your response, that might not be covered by health insurance.

Despite these hurdles, compassionate drug use does happen. The FDA receives over 1,000 individual use applications per year, and approves over 99% of those requests.

Expanded access programs (EAPs)

There are 3 types of EAPs:

In w﻿idespread (large-scale) use EAPs, a company sponsoring an unapproved drug in the late stages of drug development can offer expanded access for patients who are not able to enroll in a clinical trial. These EAPs are typically managed by the company sponsoring the drug.

In intermediate (medium-size) use EAPs,a company sponsoring an unapproved drug may offer expanded access to groups of patients during early or late stages of drug development, and usually manages the EAP.

Inindividual use EAPs,an individual patient may be able to apply for access to an unapproved drug if they don't quality for an existing clinical trial, widespread use EAP, or intermediate use EAP. In this type of expanded access, the patient's doctor must ask the drug company if the drug can be used for the patient and find out if the drug company will supply it. If the company agrees, the doctor works with the drug company to ask the FDA to approve the drug for use by this one patient. The FDA requires the doctor to send information about the patient, why the request is being made, and the proposed treatment plan. The FDA also asks that the doctor get a signed informed consent from the patient. (See Informed Consent for more on this.) The time this process takes varies, but if it’s an emergency, the FDA can complete the paperwork in 24 hours.

﻿In general, EAPs are for patients who meet all of these conditions:

Have a serious and life-threatening condition

Are not eligible for any current clinical trial that's using the drug

Have no other comparable treatment options

Are likely to have benefits that outweigh the risks involved

In addition, granting patients the use of a drug through an EAP should not interfere with ongoing clinical trials of the drug. The FDA approves or denies EAP requests based on available reports from the company sponsoring the drug in clinical trials, including safety information and data about how the drug may treat cancer. To learn more, see the FDA's information about EAPs.

"Right to Try"

A federal law passed in 2018 allows patients another path to access unapproved drugs, without needing the review and approval of the FDA. This pathway is commonly referred to as "Right to Try." "Right to Try" laws do not replace EAPs, but provide another way to access unapproved drugs.

It's important to understand that "Right to Try" does not actually give patients the right to try any unapproved drug they wish to try. Instead, it gives them the right to request access to an unapproved drug from the company that makes it, without having to go through the FDA.

Bypassing the FDA does not necessarily mean that such access will be granted. There are a number of reason access may be denied, including:

If the patient does not meet the criteria spelled out in the law

If the drug company refuses to provide the drug, of if they aren't able to make enough of it for all of the people requesting it

Bypassing the FDA also means there is potentially less oversight for exactly how the drug is being given.

To be eligible for "Right to Try," a person must:

Be diagnosed with a life-threatening disease or condition

Have tried all approved treatment options for the disease or condition

Be certified by a doctor that they are unable to participate in a clinical trial for the investigational drug

Give written informed consent that they understand the risks of taking the investigational drug

In addition, the drug itself must have already been through a phase I clinical trial. (This is the earliest phase of clinical trials, which is generally intended to start looking at the safety of the drug and the proper dose to use.)

How are EAPs and Right to Try different? How are they the same?

There are some differences between EAP and Right to Try pathways. Some of these might be good or bad, depending on the situation.

EAPs

Right to Try

Specific criteria are required for patients to get access to a drug being studied, such as:

The patient must have a life-threatening condition

The drug must be being tested in clinical trials

The patient isn’t eligible for clinical trials of the drug

Yes

Yes

FDA approval for access to a drug being studied is required.

Yes

No

Request for drug access must be made by a licensed doctor.

Yes

Yes

Includes some oversight about drug safety by the FDA and the treatment center’s institutional review board (IRB)

Yes

No

Drug and clinical trial sponsors can decline requests for various reasons.

Yes

Yes

If access is approved, insurance companies are required to pay the cost of drug and associated administration/treatment.

No

No

Cost charged to a patient is limited to direct drug costs, although sometimes indirect charges to the patient are possible.

Yes

Yes

Adverse events must be immediately reported to FDA.

Yes

No

What should I ask my doctor about compassionate drug use?

Here are some questions you may want to ask if your doctor is thinking about compassionate drug use:

Are there any approved treatments that I haven’t tried?

Is there any evidence to support the use of this drug to treat my type of cancer?

What makes you think this drug could help me?

In what way do you think this drug is likely to work better than an approved drug?

What are the known risks and benefits of treatment with this drug?

Will the drug company give me the drug for free? If not, how is it to be paid for? What costs will I have to pay to get the drug? Will my insurance cover any costs?

Should we consider expanded access (EAP) or a "Right to Try" path? What are the pros and cons of each?

What will we have to do to get access to this drug?

How long do you think it will take for me to get access to this drug?

Where do we start if I want to apply for compassionate drug use?

Your doctor or one of the office staff will work with you on this process.

Applying through an expanded access program (EAP)

If you and your doctor find a drug you want to get access to through an EAP, your doctor will need to contact the drug company to see if they would be willing to provide the drug as part of your treatment. If so, the next step is to contact the FDA's Center for Drug Evaluation and Research (CDER) at 1-301-796-3400 or druginfo@fda.hhs.gov, or the after-hours FDA Emergency Call Center at 1-866-300-4374 (toll free). See the FDA's information on EAPs to learn more.

Applying through "Right to Try"

For this pathway, your doctor will first need to contact the drug company to find out if you might be able to access the drug. (As a patient, you might be able to make the initial contact with the company to find out about your eligibility or if the drug is available, but the request for the drug will need to come from your doctor.) Then, your doctor will need to follow certain steps and work with you to get access if the drug is available.

U.S. Food and Drug Administration (FDA). Expanded access to investigational drugs for treatment use: questions and answers. Accessed at https://www.fda.gov/downloads/drugs/guidances/ucm351261.pdf on November 16, 2018.

U.S. Food and Drug Administration (FDA). Expanded access: Information for patients. Accessed at https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/ucm20041768.htm
on November 20, 2018.

Last Medical Review: November 19, 2018 Last Revised: November 19, 2018